I don’t know about you, but when I am in DC or receive government related information, I am always about 3 acronyms behind in the conversation.  PDUFA – this is one acronym you want to put in your memory bank for 2012.  PDUFA – Prescription Drug User Fee Act.

This legislation is all about FDA, about what biotech’s/pharmaceuticals have to pay for the process, about rare disorders and incentives to develop drugs for rare conditions, and about the process of approval.  It is difficult to wade through the legislation, sometimes difficult to clearly understand the ins and outs, but this legislation impacts our world, our sons, and our hope.

The PPMD Board has developed a policy that underpins and supports this legislation.

So many good things are happening in Duchenne.  We have potentially promising new drugs in development and in trial and (cross fingers and pray) and believe that it is a new day in Duchenne, that this generation of boys will benefit from treatments.

We are learning more about Duchenne all the time.  Steroids have had significant impact, not only on improving/extending function, but on clinical variability.  We know most of the boys (not all) respond to steroids.  We see the ‘bump’ or change in function within 3-6 weeks of starting steroids.  We also know that a certain group of individuals do better than expected for reasons we could not explain, but we are learning. These individuals are referred to as ‘outliers,’ but studies on modifying genes are beginning to explain these differences, which means that trials will become more constricted, more narrow in terms of inclusion and exclusion criteria.

So why the PPMD policy?

It has been developed to state our position, to invite dialogue, and to encourage education about clinical variability, outcome, and benefit and risk.

As far as rare disorders, Duchenne is ‘out front’ in terms of possibilities.  Today, trials are underway or in development on gene correction strategies (antisense oligonucleotides – exon skipping), nonsense mutations (ataluren), negative and positive regulators of muscle (myostatin inhibition and igf-1), and compounds with the potential to replace Dystrophin (Tivorsan’s biglycan, Summit’s SMT C 1100, PTC’s utrophin compound). RADD (review of approved drugs working group) is reviewing the myriad of approved drugs that have been tested in the mdx mouse, to determine those drugs that may need additional animal studies and to establish priorities for clinical studies.   As a community, we see treatments on the horizon. We clearly see the day we will End Duchenne.

For this reason the PDUFA legislation is critical.   We need to better understand clinical variability and to keep that variability in mind as we develop clinical trials.  Maybe the single primary outcome measure such as the 6MWT (six minute walk test) is inadequate to reflect benefit across the population.  It may be worth considering a composite score, a list of outcomes that may better reflect improvement across a variable population.

We are hopeful FDA will consider conditional approval.  The idea here is that if in clinical studies a drug is demonstrated to be safe, but has not reached clinical significance with the primary outcome BUT has demonstrated some positive signs of surrogate composite measures, we believe that  the drug (or biologic) should be made available to all individuals that meet the criteria for the drug, while additional trials are conducted or post marketing data confirms efficacy.

And because we are learning more and more about individuals with Duchenne, we expect that trials will become more constricted, that is, focus on specific subsets of the population. We want to make sure the labels, the information that describes eligibility, is broad, to include all individuals with the potential to benefit.   We do not want lines drawn based on function.

Essentially, the PPMD policy was derived to inform, to accelerate approvals, and to ensure all who are eligible have access.

PDUFA.  Add this acronym to your vocabulary.  It is renewed every 5 years and this is the year.   And it matters.




Pat Furlong, Founding President, CEO
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Comment by Amy O'Connor on February 23, 2012 at 9:46am

Pat, thank you for raising awareness of this critically important issue. The reauthorization of PDUFA is essential to improving the FDA’s drug approval process, promoting the development of breakthrough treatments for disorders like Duchenne, and giving patients access to innovative new medicines. That’s why the Campaign for Modern Medicines is urging Congress to pass a clean PDUFA in a timely manner. If you’d like to learn more about PDUFA or how you can join our campaign, please visit our website at www.modernmedicines.com or email me at amy@modernmedicines.com.

Comment by Pat Furlong on February 21, 2012 at 8:26pm

Keith, agree that access for all patients is a critical factor.  We are beginning to explore this area and are learning from other groups (such as Pompe) who have been through this and working on a strategy. 

Comment by Keith Van Houten on February 21, 2012 at 8:10pm

Pat, I know conditional approvals have been granted for drugs for other conditions - Avastin for breast cancer, for instance.

What's your thoughts on insurance coverage for a conditionally approved drug?  

Cost is the immediate next hurdle to overcome.  An approved drug that you can't afford, in my opinion, is harder to deal with emotionally than no drug at all.  

Comment by Anita Bullers on February 20, 2012 at 4:27pm

Pat, thank you for helping me understand more about this important and complicated piece of legislation...PDUFA. The recent webinar was helpful also and I feel the more we learn about PDUFA,  the better job we can do advocating for our boys.  

Comment by Blazena Kaderabkova on February 20, 2012 at 10:59am

Pat, thank you very much for your effort.

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